Antisense Oligonucleotide-Mediated Correction of Transcriptional Dysregulation is Correlated with Behavioral Benefits in the YAC128 Mouse Model of Huntington's Disease

Abstract excerpt

BACKGROUND: Huntington's disease (HD) is a neurological disorder caused by mutations in the huntingtin (HTT) gene, the product of which leads to selective and progressive neuronal cell death in the striatum and cortex. Transcriptional dysregulation has emerged as a core pathologic feature in the CNS of human and animal models of HD. It is still unclear whether perturbations in gene expression are a consequence of...

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